Trial · NCT03633058

A Study to Evaluate Ketamine for the Treatment of Rett Syndrome

NCT03633058, Phase 2, Completed, Rett Syndrome Research Trust; results first posted 2024-03-27

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. A Study to Evaluate Ketamine for the Treatment of Rett Syndrome. Trials, record NCT03633058. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03633058

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: A Study to Evaluate Ketamine for the Treatment of Rett Syndrome
NCT number: NCT03633058
Status: Completed
Phases: Phase 2
Enrollment: 23 participants, actual
Lead sponsor: Rett Syndrome Research Trust
Conditions: Rett Syndrome
Interventions: Ketamine
First posted: 2018-08-16
Last updated: 2024-05-28
Results first posted: 2024-03-27

Source ClinicalTrials.gov

Registered outcomes10

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Dose-Limiting Adverse Events	6 weeks	The Number of Participants with Treatment-emergent adverse events on ketamine compared to placebo will be summarized
Other	Continuous Biosensor Data	6-8 weeks	2 different non-invasive, wearable devices will be used in the study to determine changes in physiologic measures for the patient in the home environment. Biosensors will be worn continuously during the screening and treatment period to measure activity, sleep, position, heart rate, and breathing.
Other	Clinical Global Impression of Improvement	4 weeks	Clinicians will use a 7 point Likert Scale to rate change from baseline symptom severity by addressing the question, "Compared to the patient's condition of Rett syndrome prior to treatment initiation at baseline, the patient's current condition is: 1 very much improved, 2 much improved, 3 minimally improved, 4 no change, 5 minimally worse, 6 much worse, 7 very much worse. No change would be scored a 0, while improvement would be scored at -1, -2, or -3 and worsening would be scored +1, +2 or +3, depending on the degree of perceived change. Negative change indicates improvement while positive change indicates worsening
Other	Motor Behavioral Assessment	4 weeks	a 37-item questionnaire for clinicians to evaluate current behavioral/social, orofacial/respiratory, and motor/physical symptoms. Each item is rated either 0 (normal or never), 1 (mild or rare), 2 (moderate or occasional), 3 (marked or frequent), 4 (very severe or constant), where higher numbers indicate higher severity. Each subscale is summed for a subscale score, while the total score is a sum of the subscale scores. The behavioral/social subscale score may range from 0 to 64; the orofacial/respiratory subscale may range from 0 to 28, and motor/physical subscale may range from 0 to 56. Total score may range from 0 to 148.
Other	Clinician Domain Likert Scale	4 weeks	an 8 category (domain) questionnaire to assess current hand function, walking, verbal and non-verbal communication, comprehension, attention, behavior problems, and mood by considering the question, "Considering your experience with the patient at this visit, please rate the level of function in each category". Each domain will be rated on a 7 point Likert Scale where the clinician will select 1 of 7 choices: 1 normal, 2 borderline abnormal, 3 mildly abnormal, 4 moderately abnormal, 5 markedly abnormal, 6 severely abnormal, 7 extremely abnormal. Lower values indicate lesser severity while higher values indicate higher severity.
Other	Rett Syndrome Behaviour Questionnaire	4 weeks	a 45-item questionnaire for clinicians to evaluate current behavior and emotional features. Each item is rated either 0 (not true or not done), 1 (somewhat or sometimes true) and 2 (very true), where higher numbers indicate higher severity. Total score is summed and may range from 0 to 90.
Other	Parent Domain Likert Scale	4 weeks	a 9 category (domain) questionnaire to assess current hand function, walking, verbal and non-verbal communication, comprehension, attention, behavior problems, mood, and seizure activity, by considering the question, "Considering your experience with your child over the past 7 days, please rate your child's level of function in each category". Each domain will be rated on a 7 point Likert Scale where the parent/caregiver will select 1 of 7 choices: 1 normal, 2 borderline abnormal, 3 mildly abnormal, 4 moderately abnormal, 5 markedly abnormal, 6 severely abnormal, 7 extremely abnormal. Lower values indicate lesser severity while higher values indicate higher severity.
Other	Children's Sleep Habits Questionnaire	4 weeks	a 35-item questionnaire for parents to evaluate current sleep and common sleep problems. The parent/caregiver will rate each item as 1 (rarely), 2 (sometimes), or 3 (usually), where higher scores indicate higher severity. The parent/caregiver will also indicate if the item is a problem or not. A total score of the sum of each item and 8 subscale scores (bedtime resistance, sleep onset delay, sleep duration, sleep anxiety, night wakings, parasomnias, sleep disordered breathing, and daytime sleepiness) are possible. Total scores between 0 and 70 are possible, where higher scores indicate more sleep problems.
Other	Rett Caregiver Burden Inventory Assessment	4 weeks	a 26-item questionnaire for parents to evaluate the burden of care on their quality of life. The parent/caregiver will rate each item as 0 (never), 1 (rarely), 2 (sometimes), 3 (quite frequently), or 4 (nearly always) where higher scores indicate higher severity. Total scores between 0 and 104 are possible, where higher scores indicate more caregiver burden.
Other	EEG Signature	Day 1, Day 15	ketamine EEG alpha, beta, gamma, delta and theta waveform signatures compared to placebo to indicate target engagement

Source ClinicalTrials.gov

Outcomes10

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Dose-Limiting Adverse Events	6 weeks	The Safety Population consisted of all patients who received at least 1 dose of study treatment.	Participants	COUNT_OF_PARTICIPANTS
OTHER_PRE_SPECIFIED	Continuous Biosensor Data	6-8 weeks
OTHER_PRE_SPECIFIED	Clinical Global Impression of Improvement	4 weeks
OTHER_PRE_SPECIFIED	Motor Behavioral Assessment	4 weeks
OTHER_PRE_SPECIFIED	Clinician Domain Likert Scale	4 weeks
OTHER_PRE_SPECIFIED	Rett Syndrome Behaviour Questionnaire	4 weeks
OTHER_PRE_SPECIFIED	Parent Domain Likert Scale	4 weeks
OTHER_PRE_SPECIFIED	Children's Sleep Habits Questionnaire	4 weeks
OTHER_PRE_SPECIFIED	Rett Caregiver Burden Inventory Assessment	4 weeks
OTHER_PRE_SPECIFIED	EEG Signature	Day 1, Day 15

Source ClinicalTrials.gov

Results by group20

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Units
Dose-Limiting Adverse Events	0.75 mg/kg Ketamine	TEAEs	2	Participants
Dose-Limiting Adverse Events	0.75 mg/kg Placebo	TEAEs	4	Participants
Dose-Limiting Adverse Events	1.5 mg/kg Ketamine	TEAEs	6	Participants
Dose-Limiting Adverse Events	1.5 mg/kg Placebo	TEAEs	3	Participants
Dose-Limiting Adverse Events	0.75 mg/kg Ketamine	SAEs	0	Participants
Dose-Limiting Adverse Events	0.75 mg/kg Placebo	SAEs	0	Participants
Dose-Limiting Adverse Events	1.5 mg/kg Ketamine	SAEs	0	Participants
Dose-Limiting Adverse Events	1.5 mg/kg Placebo	SAEs	0	Participants
Dose-Limiting Adverse Events	0.75 mg/kg Ketamine	AEs leading to discontinuation	0	Participants
Dose-Limiting Adverse Events	0.75 mg/kg Placebo	AEs leading to discontinuation	0	Participants

1 to 10 of 20Source ClinicalTrials.gov

See all 20 results by group

Participant flow12

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	0.75 mg/kg (Placebo First, Then 0.75 mg/kg Ketamine)	5
Overall Study	STARTED	0.75 mg/kg (0.75 mg/kg Ketamine First, Then Placebo)	6
Overall Study	STARTED	1.5 mg/kg (Placebo First, Then 1.5 mg/kg Ketamine)	6
Overall Study	STARTED	1.5 mg/kg (1.5 mg/kg Ketamine First, Then Placebo)	6
Overall Study	COMPLETED	0.75 mg/kg (Placebo First, Then 0.75 mg/kg Ketamine)	5
Overall Study	COMPLETED	0.75 mg/kg (0.75 mg/kg Ketamine First, Then Placebo)	6
Overall Study	COMPLETED	1.5 mg/kg (Placebo First, Then 1.5 mg/kg Ketamine)	5
Overall Study	COMPLETED	1.5 mg/kg (1.5 mg/kg Ketamine First, Then Placebo)	6
Overall Study	NOT COMPLETED	0.75 mg/kg (Placebo First, Then 0.75 mg/kg Ketamine)	0
Overall Study	NOT COMPLETED	0.75 mg/kg (0.75 mg/kg Ketamine First, Then Placebo)	0

1 to 10 of 12Source ClinicalTrials.gov

See all 12 participant flow

Adverse events84

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Constipation	Gastrointestinal disorders	0.75 mg/kg Ketamine	0	11	0
other	Constipation	Gastrointestinal disorders	0.75 mg/kg Placebo	1	11	1
other	Constipation	Gastrointestinal disorders	1.5 mg/kg Ketamine	0	12	0
other	Constipation	Gastrointestinal disorders	1.5 mg/kg Placebo	1	12	1
other	Diarrhoea	Gastrointestinal disorders	0.75 mg/kg Ketamine	0	11	0
other	Diarrhoea	Gastrointestinal disorders	0.75 mg/kg Placebo	0	11	0
other	Diarrhoea	Gastrointestinal disorders	1.5 mg/kg Ketamine	1	12	1
other	Diarrhoea	Gastrointestinal disorders	1.5 mg/kg Placebo	0	12	0
other	Eructation	Gastrointestinal disorders	0.75 mg/kg Ketamine	0	11	0
other	Eructation	Gastrointestinal disorders	0.75 mg/kg Placebo	0	11	0

1 to 10 of 84Source ClinicalTrials.gov

See all 84 adverse events

Adverse event totals12

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	0.75 mg/kg Ketamine	0	11
Total, all-cause mortality	0.75 mg/kg Placebo	0	11
Total, all-cause mortality	1.5 mg/kg Ketamine	0	12
Total, all-cause mortality	1.5 mg/kg Placebo	0	12
Total, other adverse events	0.75 mg/kg Ketamine	2	11
Total, other adverse events	0.75 mg/kg Placebo	4	11
Total, other adverse events	1.5 mg/kg Ketamine	7	12
Total, other adverse events	1.5 mg/kg Placebo	3	12
Total, serious adverse events	0.75 mg/kg Ketamine	0	11
Total, serious adverse events	0.75 mg/kg Placebo	0	11

1 to 10 of 12Source ClinicalTrials.gov

See all 12 adverse event totals

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
39856538	DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT03633058

GET https://conductscience.org/api/trials/v1/NCT03633058/events?page=2

API reference For AI assistants